Hydroxyurea + EPO for Sickle Cell Disease

(ACHiEvE-SCD Trial)

Recruiting at 1 trial location

Overseen By Jude C Jonassaint, BSN

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Julia Xu

Must be taking: Hydroxyurea

Must not be taking: Voxelotor, EPO

Disqualifiers: Chronic transfusion, Iron deficiency, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

17 Participants Needed

Trial Summary

What is the purpose of this trial?

The proposed study is a Phase 1/2 multi-center study evaluating the safety and efficacy of erythropoietin (EPO) in combination with hydroxyurea in the treatment of chronic anemia in patients with sickle cell disease (SCD).

Research Team

Julia Z Xu, MD, MScGH

Principal Investigator

University of Pittsburgh

Eligibility Criteria

Adults over 18 with confirmed sickle cell disease (HbSS or HbS/β0-thalassemia), not on blood transfusion programs, and without recent use of certain drugs like voxelotor or EPO. Participants must have been on a stable dose of hydroxyurea for at least 60 days and plan to continue it during the trial.

Inclusion Criteria

Screening transferrin saturation ≥ 20% and ferritin ≥ 100 ng/mL

Screening Hb ≤ 9.0 g/dL

I have been on a consistent dose of hydroxyurea for the last 60 days and plan to continue.

Exclusion Criteria

I have had very high blood pressure on two separate tests.

I have had a blood clot in the last 6 months.

I have not had a serious illness, infection, or pain crisis in the last 2 weeks.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants on stable hydroxyurea therapy receive increasing doses of subcutaneous erythropoietin (EPO) for 12 weeks to assess safety and efficacy.

12 weeks

Regular visits for monitoring and dose adjustments

Extended Treatment

Participants may continue EPO treatment for an additional 12 weeks as clinically indicated, with further assessment of endpoints.

12 weeks

Regular visits for monitoring and assessment

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Epoetin Alfa
• Hydroxyurea

Trial Overview The study is testing the combination of erythropoietin (EPO) with hydroxyurea to treat chronic anemia in sickle cell disease patients. It's a Phase 1/2 trial, meaning they're looking at safety and how well it works across multiple centers.

Participant Groups

Group I: Erythropoietin Experimental Treatment 2 Interventions

Subjects on a stable dose of hydroxyurea will be treated with increasing doses of subcutaneous erythropoietin (EPO) as tolerated for an initial 12 weeks, during which the main safety and efficacy endpoints (including the primary endpoint of hemoglobin response) will be assessed. Subjects may continue on treatment for an additional 12 weeks as clinically indicated, with assessment of additional endpoints at the end of the 24-week study period.

Hydroxyurea is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Hydroxyurea for:

• Sickle cell disease
• Chronic myeloid leukemia
• Solid tumors
• Thrombocythemia

Approved in European Union as Hydroxycarbamide for:

• Sickle cell syndrome
• Chronic myeloid leukaemia
• Essential thrombocythaemia
• Polycythaemia vera

Approved in Canada as Hydroxyurea for:

• Sickle cell disease
• Chronic myeloid leukemia
• Thrombocythemia

Who Is Running the Clinical Trial?

Julia Xu

Lead Sponsor

Trials

1

Recruited

20+

Carnegie Mellon University

Collaborator

Trials

80

Recruited

540,000+

American Society of Hematology

Collaborator

Trials

14

Recruited

20,800+